OBJECTIVES

Management of hemophilia A (HA) combine two main approaches: a preventative one where medicines are used to prevent bleeding and joint and muscle damage and an on-demand treatment, where medicines aim to treat prolonged bleeding. One objective of the HEMRAUDE study was to describe patients with HA and HA care practices in real life in France.

METHODS

A retrospective analysis of all HA male patients figuring in the National French healthcare claims database SNDS between January 1, 2016 and December 31, 2017 was conducted. Using treatment algorithm and expert review of patient’s files, patients were classified in 6 treatment groups: no treatment, on-demand FVIII, FVIII immune tolerance induction (ITI) protocol, FVIII prophylaxis, on-demand bypassing agents, and bypassing agent prophylaxis. Patterns of treatment were described including drug utilization, physician and paramedics services, hospitalizations.

RESULTS

Overall, 4,172 patients (all degree of severity) were included in the analysis, aged on average 35.2 years, 34.1% were less than 18 years old. Based on a treatment algorithm, 2.8% had FVIII inhibitors in 2017. Half of the patients (51.6%) did not receive any treatment for HA over this year, 25% were treated on demand, and 20.6 % had long-term prophylaxis (30.3% under 18 y). Only 1.6% were treated with by-passant agents either on demand or in prophylaxis. Overall, 8.2% have received at least once strong opioids over the year and 5.0% desmopressin. Only one half of the patients have visited a specialist over the year (55.8%). Finally, 26.7% of patients were hospitalized at least once and 19.8% had to go to the emergency room.

CONCLUSIONS

A wide-range integrated care management of HA was observed in 2017 in real-life. Few patients have developed FVIII inhibitors. A rather small percentage of patients including children received prophylaxis which may call into question the HA guidelines follow-up in France.